A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

Launched by EPICRISPR BIOTECHNOLOGIES, INC. · Mar 26, 2025

Keywords

ClinConnect Summary

This clinical trial is researching a new treatment called EPI-321 for adults with facioscapulohumeral muscular dystrophy (FSHD), a condition that affects muscle strength. The main goal of the study is to determine how safe EPI-321 is and if it shows any early signs of helping patients. Participants in this trial must be adults aged 18 to 75 who have been diagnosed with FSHD Type 1 and have a specific level of muscle weakness. They also need to have good liver and kidney function.

If someone qualifies and decides to participate, they will receive a single dose of EPI-321 through an IV in a hospital setting. After this, they will have regular check-ups and tests at the clinic for about 5 years to monitor their health and any effects of the treatment. It’s important to note that this trial is not yet recruiting participants, but it aims to provide valuable insights into the safety and potential effectiveness of EPI-321 for those living with FSHD.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Able and willing to provide informed consent
• • Male or female 18 to 75 years of age
• • Clinical diagnosis of FSHD with genetic Type 1
• • FSHD Ricci clinical severity score 2 to 4 (on 5-point scale)
• • Has adequate liver function
• • Has adequate kidney function
• Exclusion Criteria:
• • Has an anti-AAVrh74 total binding antibody titer \> 1:400
• • Requires a walker or wheelchair for ambulation
• • Pregnant and/or breastfeeding at baseline or is planning to become pregnant during the first 12 months following EPI-321 administration
• • Has FSHD Type 2
• • Has a concurrent or past medical conditions could jeopardize the safety of the participant